~ Completed Target Patient Dosing in Phase III HOPE-B Trial of Etranacogene Dezaparvovec in Hemophilia B ~~ Announced 22 Presentations at Upcoming ASGCT Virtual Annual Meeting ~LEXINGTON, Mass. and AMSTERDAM, April 29, 2020 (GLOBE NEWSWIRE) — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2020 and highlighted recent progress across its business.“Despite the ongoing global crisis caused by the coronavirus pandemic, we ended the first quarter of 2020 in a position of strength and remain well capitalized,” stated Matt Kapusta, chief executive officer of uniQure. “I am very proud of the continued dedication of our employees in both the United States and the Netherlands, and thank them for the outstanding work they continue to do in driving forward our clinical programs, research pipeline, and manufacturing and technology operations. We are well positioned for an important second half of the year when we expect to announce new clinical data and advancements from across the business.”Recent Company Progress• Advancing late-stage development of etranacogene dezaparvovec (AMT-061) for the treatment of hemophilia B In the first quarter, the Company announced the achievement of targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. A total of 54 patients now have received the one-time dose of etranacogene dezaparvovec.
The Company remains on track to provide 26-weeks of Factor IX data for all 54 patients in the HOPE-B trial before the end of this year and to file an application for marketing authorization in 2021.
The manufacturing process validation for etranacogene dezaparvovec is ongoing, and the Company believes it is able to produce material at commercial scale.• Advancing AMT-130 into clinical development for the treatment of Huntington’s disease
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